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基于模型的方法来支持肥胖患者的药物开发:监管视角
Xiaolei Pan1, Li Wang2, Jiang Liu1
1Division of Pharmacometrics, Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA.
基于模型的药物开发 (MIDD) 有助于了解肥胖如何影响药物的有效性. 对于优化药物开发和对肥胖患者的监管决策,MIDD方法至关重要.
科学领域:
- 药理学和药物开发领域
- 临床试验设计 临床试验设计
- 肥胖问题研究研究
背景情况:
- 肥胖 (BMI ≥30 kg/m2) 是一个主要的健康问题,与许多相关疾病和增加的死亡率有关.
- 肥胖的高患病率需要在临床试验设计中进行专门考虑.
- 了解肥胖对药理动力学 (PK),药理动力学 (PD) 和药物疗效的影响是具有挑战性的,因为肥胖患者的早期招募有限.
研究的目的:
- 审查肥胖对药物PK,PD和疗效的影响.
- 提供模型信息化药物开发 (MIDD) 应用在治疗肥胖的药物开发中的概述.
- 突出MIDD在对肥胖患者群体的监管决策中的作用.
主要方法:
- 文献综述总结了肥胖对药物行为和临床结果的影响.
- 对用于肥胖和相关疾病的药物开发计划中使用的MIDD方法的分析.
- 检查证实MIDD在剂量选择和有效性评估中的有用性的案例研究.
主要成果:
- 肥胖显著影响药物PK,PD和疗效,需要量身定制的方法.
- MIDD有效地整合了各种数据,为肥胖个体的临床药物开发提供了信息.
- MIDD支持关键的监管决策,包括剂量优化和寻找新适应症.
结论:
- MIDD是设计临床试验和为肥胖患者做出监管决策的宝贵工具.
- 在肥胖人群的特定剂量场景中,MIDD促进了PK,PD和疗效的估计.
- 案例示例显示MIDD在支持剂量选择和确认肥胖患者的有效性方面的承诺,包括儿科.
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